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Results for “Dysostosis Multiplex”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Study completedNCT06406153
What this trial is testing

Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients

Who this might be right for
Mucopolysaccharidosis Type 1
Cinnagen 12
Testing effectiveness (Phase 2)Ended earlyNCT01372228
What this trial is testing

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Who this might be right for
Hurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more
Talaris Therapeutics Inc. 3
Early research (Phase 1)Looking for participantsNCT04532047
What this trial is testing

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Who this might be right for
MPS IMPS IIMPS IVA+6 more
University of California, San Francisco 10
Not applicableEnded earlyNCT04008849
What this trial is testing

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 3
Not applicableWithdrawnNCT00286689
What this trial is testing

Effects of Growth Hormone in Chronically Ill Children

Who this might be right for
Hurler SyndromeCerebral PalsyJuvenile Rheumatoid Arthritis+2 more
University of Texas Southwestern Medical Center
Testing effectiveness (Phase 2)WithdrawnNCT05665036
What this trial is testing

Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy

Who this might be right for
MPS IMPS1-SMPS1-HS
Sigilon Therapeutics, Inc.
Not applicableLooking for participantsNCT07361536
What this trial is testing

Cardiac Structure and Function in MPS

Who this might be right for
MPS IMPS IV AMPS - Mucopolysaccharidosis+1 more
Children's Hospital of Orange County 240
Not applicableEnded earlyNCT01572636
What this trial is testing

Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

Who this might be right for
Mucopolysaccharidosis Type IHMPS IHurler Syndrome
Masonic Cancer Center, University of Minnesota 20
Large-scale testing (Phase 3)Study completedNCT00146770
What this trial is testing

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 45
Early research (Phase 1)Ended earlyNCT00215527
What this trial is testing

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

Who this might be right for
Mucopolysaccharidosis ILysosomal Storage DiseasesSpinal Cord Compression
Patricia I. Dickson, M.D. 4
Not applicableUnknownNCT04958070
What this trial is testing

The Intensively Follow-up Examinations for Asymptomatic MPS I Infants in Taiwan

Who this might be right for
MPS I
Mackay Memorial Hospital 20
Testing effectiveness (Phase 2)WithdrawnNCT02715505
What this trial is testing

Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)

Who this might be right for
Inherited Metabolic Disorders IMD
Novartis Pharmaceuticals
Testing effectiveness (Phase 2)Study completedNCT00730314
What this trial is testing

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Who this might be right for
Sickle Cell DiseaseThalassemiaAnemia+9 more
Children's Hospital Los Angeles 25
Testing effectiveness (Phase 2)Study completedNCT00176891
What this trial is testing

Stem Cell Transplant w/Laronidase for Hurler

Who this might be right for
Mucopolysaccharidosis IHurler Syndrome
Masonic Cancer Center, University of Minnesota 25
Not applicableStudy completedNCT00144794
What this trial is testing

Mucopolysaccharidosis I (MPS I) Registry

Who this might be right for
Mucopolysaccharidosis I (MPS I)
Genzyme, a Sanofi Company 1,406
Not applicableStudy completedNCT00852358
What this trial is testing

Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

Who this might be right for
Cognitive DeclineMucopolysaccharidosis IHurler-Scheie Syndrome+2 more
Patricia I. Dickson, M.D. 9
Post-approval studies (Phase 4)Ended earlyNCT00418821
What this trial is testing

The Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 2
Testing effectiveness (Phase 2)Ended earlyNCT00668564
What this trial is testing

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Who this might be right for
Hurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more
Masonic Cancer Center, University of Minnesota 18
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104
What this trial is testing

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
Not applicableStudy completedNCT01873911
What this trial is testing

Neurobehavioral Phenotypes in MPS III

Who this might be right for
Sanfilippo Syndrome Type ASanfilippo Syndrome Type BHurler Syndrome
University of Minnesota 30
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